News and press releases about the Joshua Frase Foundation and its efforts behind CNM/MTM

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Wed, Sep 18, 2019

The importance of patient registries. Recently, the Joshua Frase Foundation's patient registry was used in collaboration in this published paper, RECENSUS.

Mortality and respiratory support in X-linked myotubular myopathy: a RECENSUS retrospective analysis
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Tue, May 07, 2019

How this promising gene therapy for a rare neuromuscular disease was fueled by passionate parents and a dog

Many treatments for rare diseases begin with families who work tirelessly, sometimes for decades, to fund the initial studies leading to the clinic. For X-linked Read More

Wed, May 01, 2019

Audentes Therapeutics Presents New Positive Data from ASPIRO, the Phase 1/2 Clinical Trial of AT132 for X-linked Myotubular Myopathy, at 22nd Annual Meeting of the American Society of Gene and Cell Therapy

New data include 48 weeks of follow-up for six treated patients in dose Cohort 1 and 24 weeks for three treated patients in dose Cohort 2 ...
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Tue, Apr 30, 2019

Dynacure Announces Approval of Clinical Trial Application for DYN101, an Antisense Medicine to Treat Rare Disease ‘Centronuclear Myopathies’

Company expects to initiate Phase 1 / 2 study 'Unite-CNM' in 2H2019 Strasbourg (France), April 30, 2019 Dynacure, a...
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Mon, Jul 09, 2018

The Matriarch of the First MTM Dog Colony...The Power of Patient Advocacy

The Joshua Frase Foundation (JFF) is a non-profit organization founded with a simple yet seemingly unattainable vision: to find a cure for Myotubular Myopathy (MTM). The...
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Thu, Jun 21, 2018

Rare Leader: Alison Rockett Frase, President of the Joshua Frase Foundation

The Basics Name : Alison Rockett Frase Title : President, co-founder, and patient advocate ...
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Thu, Jun 07, 2018

Gene Therapy for Myotubular Myopathy: Early Signs of Success!

Parents cherish developmental milestones, from a newborn’s grip of an offered finger; to an infant’s holding her head up the first time; to rolling over, creeping, and...
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Fri, Jun 01, 2018

From Canines to a Cure

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Wed, Feb 28, 2018

A Boy With the Will to Live: Biobanking Contributing to Breakthroughs in Rare Diseases

Joshua Frase was born on February 2, 1995. When Joshua was three months old, a muscle biopsy revealed that he had the ultra-rare X-linked myotubular myopathy (XLMTM), a...
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Wed, Nov 22, 2017

UW, Family Rewriting Neuromuscular Research

Physicians and researchers at the UW Medicine Institute for Stem Cell and Regenerative Medicine have partnered up with a nonprofit foundation in one of the leading efforts to...
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Tue, Oct 17, 2017

Transnetyx produces "Brick in the Wall," second short film in its Love Letter Series

MEMPHIS, Tenn., Oct. 17, 2017 /PRNewswire/ -- Transnetyx, an automated genotyping company that serves biomedical researchers worldwide, today announced the premier of the...
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Wed, Oct 11, 2017

The Joshua Frase Foundation for Congenital Myopathy Research, Inc.

Article on Alison's speech at the National Animal Interest Alliance, October 2017
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Thu, May 18, 2017

KIRO 7 Investigates Revolutionary Treatment in Dogs to be Tested in Children

Tue, Mar 21, 2017

Determined Parents are Moving the Needle on Gene Therapy

Families of patients are starting advocacy groups, raising money for research, and founding biotech companies to advance cures for rare diseases.
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Fri, Feb 03, 2017

Local Foundation Makes Headway In Gene Replacement Therapy

One little boy, who was granted just 15 years on this earth, touched a lot of people for the good.
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Thu, Jan 26, 2017

An Active Philanthropist

An active philanthropist, Costa is an avid supporter and board member of various charitable organizations, such as the Multiple Sclerosis Foundation; Joshua Frase Foundation,...
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Wed, Nov 23, 2016

A Walk to Remember

Students at Landrum Middle School in Ponte Vedra Beach participated in the ninth annual Walk of Remembrance on Monday. The purpose of the event was to allow students to honor...
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Sat, Nov 12, 2016

Pine Brook Resident Advocates for Patients with Rare Muscle Disease

MONTVILLE, NJ – When a family member is diagnosed with a debilitating condition, it’s natural to reach out to not only family, but others whose loved ones are going through...
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Mon, Nov 07, 2016

Odessa Mom Spreads Awareness of Her Son's Fatal Muscle Disease

Lindsay Jones and Ryan Jones are married. Ryan is an Odessa firefighter and Lindsay is a stay-at-home mom, but what she does, requires 24 hour love and care, for little...
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Mon, Oct 17, 2016

Paul Frase remembers former teammate Dennis Byrd

His post football-playing travels landed former Jets defensive tackle Paul Frase in Oklahoma City about eight or nine years ago for business, and Frase says now that he...
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Our recent campaign to increase participation in our Family Registry was published in MDA Quest Magazine, and can be seen below.